Muscular Dystrophies

CMD Research: Promising Leads

Recently published findings from two independent groups have suggested possible treatment pathways for the merosin-deficient and integrin-deficient forms of congenital muscular dystrophy (CMD).

Doxycycline fights cell death and lessens disease severity in merosin-deficient mice

Flying High ... From a Wheelchair

Flying automobiles … flying lawnmowers … flying doghouses … where’s this madness going to end?

If Chris “Lucky” Carnes has his druthers, the sky’s the limit.

For the past four years, the 33-year-old from Chase City, Va., has been busy practically every weekend with the rapidly growing hobby/sport of model aviation.

MMD Research: Drug Development?

The identification of small molecules that can block the genetic defect that causes type 1 myotonic dystrophy (MMD1, or DM1) may be the first step toward developing a new drug treatment for the disease, say researchers at the University of Rochester (N.Y.) Medical Center (URMC).

The abnormality that underlies MMD1 is a stretch of genetic material derived from DNA on chromosome 19 that contains more than the usual number of a repeating chemical sequence known as a CUG (cytosine, uracil, guanine) triplet repeat.

Speeding the Course of Clinical Trials

Trial Results in DMD Heralded as 'Proof of Concept'

The Dec. 27 issue of the prestigious New England Journal of Medicine featured encouraging results of a phase 1 clinical trial of an exon skipping compound in four boys with Duchenne muscular dystrophy (DMD). Preliminary findings from this trial, conducted in the Netherlands, were announced in May (see Progress Reports).

First US Trial of DMD Gene Therapy Under Way

The first U.S. human gene therapy trial directed at Duchenne muscular dystrophy (DMD) was launched yesterday at Columbus (Ohio) Children's Hospital, the Muscular Dystrophy Association (MDA), Children’s Hospital, and Asklepios Biopharmaceutical Inc. (AskBio) announced today. 

Neurologist Jerry Mendell administered an injection of AskBio’s Biostrophin, which contains a functional gene for the muscle protein dystrophin, into the biceps of Andrew Kilbarger, 8, of Lancaster, Ohio.

Pages